Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes-Reference-Cited by-同舟云学术

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Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

Published:2007-08-27 Issue:22 Volume:16 Page:2693-2702
ISSN:1460-2083
Container-title:Human Molecular Genetics
language:en
Short-container-title:

Author:

Fraldi Alessandro,Hemsley Kim,Crawley Allison,Lombardi Alessia,Lau Adeline,Sutherland Leanne,Auricchio Alberto,Ballabio Andrea,Hopwood John J.

Publisher

Oxford University Press (OUP)

Subject

Genetics(clinical),Genetics,Molecular Biology,General Medicine

Link

http://academic.oup.com/hmg/article-pdf/16/22/2693/13928413/ddm223.pdf

Reference42 articles.

1. Lysosomal disease;Suzuki,2002

2. The mucopolysaccharidoses;Neufeld,2001

3. Prevalence of lysosomal storage disorders;Meikle;JAMA,1999

4. Sulfamidase deficiency in a family of Dachshunds: a canine model of mucopolysaccharidosis IIIA (Sanfilippo A);Fischer;Pediatr. Res.,1998

5. Mucopolysaccharidosis IIIA (Sanfilippo syndrome) in a New Zealand Huntaway dog with ataxia;Jolly;N. Z. Vet. J.,2000

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